On August 11, 2025, Stoke Therapeutics, Inc. and Biogen Inc. announced that the first patient has been dosed in the global Phase 3 EMPEROR study of zorevunersen for the treatment of Dravet syndrome. Zorevunersen is an investigational antisense oligonucleotide (ASO) that has the potential to be the first disease-modifying treatment for this rare genetic epilepsy.
The EMPEROR study is a global, double-blind, sham-controlled clinical trial designed to evaluate the efficacy and safety of zorevunersen compared to a sham comparator over a 52-week treatment period. The study is enrolling children aged 2 to less than 18 years with Dravet syndrome, a condition characterized by refractory seizures and neurodevelopmental impairments.
The initiation of dosing in this pivotal Phase 3 study is a critical milestone in zorevunersen's development. Previous Phase 1/2 and open-label extension studies have provided data supporting substantial and durable reductions in seizures and continuing improvements in cognition and behavior, reinforcing the belief that zorevunersen may improve outcomes for patients by addressing the underlying genetic cause of Dravet syndrome.
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